GC1134A

GC1134A is a recombinant human alpha-galactosidase A (rhGLA) enzyme replacement therapy (ERT) being developed by GC BioPharma (formerly Green Cross) for the treatment of Fabry disease. Fabry disease is a rare, X-linked lysosomal storage disorder caused by mutations in the GLA gene, which leads to a deficiency of the alpha-galactosidase A enzyme. This deficiency results in the systemic accumulation of glycosphingolipids, primarily globotriaosylceramide (Gb3), within lysosomes, causing progressive damage to multiple organs, including the kidneys, heart, and nervous system. GC1134A is designed to restore enzymatic activity, facilitating the breakdown of accumulated Gb3 and potentially slowing or preventing disease progression. The candidate is currently being evaluated in Phase 1/2 clinical trials to assess its safety, tolerability, pharmacokinetics, and pharmacodynamics in patients with Fabry disease.

Other names

recombinant human alpha-galactosidase ArhGLA